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Antisense nucleic acids

  • US 10,144,931 B2
  • Filed: 09/15/2016
  • Issued: 12/04/2018
  • Est. Priority Date: 09/15/2015
  • Status: Active Grant
First Claim
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1. An antisense oligomer consisting of a nucleotide sequence selected from the group consisting of SEQ ID NOs:

  • 25, 30, 33, 79, and 80, or pharmaceutically acceptable salt or hydrate thereof,wherein the antisense oligomer is a morpholino oligomer, a peptide nucleic acid (PNA), or an oligonucleotide comprising at least one nucleotide having;

    (i) a modified sugar moiety, wherein the 2′

    -OH group of a ribose is replaced by any one selected from the group consisting of R, R′

    OR, SH, SR, NH2, NHR, NR2, N3, CN, F, Cl, Br and I (wherein R is an alkyl or an aryl and R′

    is an alkylene), or(ii) a modified phosphate-binding region selected from the group consisting of a phosphorothioate bond, a phosphorodithioate bond, an alkylphosphonate bond, a phosphoramidate bond, and a boranophosphate bond.

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